Actuate Announces Completion of Enrollment in a Phase 2 Trial of Elraglusib in Combination with FOLFIRINOX and Losartan in Patients with Previously Untreated Metastatic Pancreatic Cancer

Rhea-AI Summary

Actuate Therapeutics (NASDAQ: ACTU) announced the completion of patient enrollment in its Phase 2 trial of elraglusib combined with FOLFIRINOX and losartan for untreated metastatic pancreatic ductal adenocarcinoma (mPDAC). The trial, led by Dr. Colin Weekes at Massachusetts General Hospital and supported by the Lustgarten Foundation, enrolled 56 treatment-naïve mPDAC patients. Initial data presented at the AACR Special Conference in September 2024 showed early evidence of enhanced clinical activity. The primary objectives are to assess safety, tolerability, and progression-free survival. Final results are expected in 2026.

CEO Daniel Schmitt highlighted the milestone and noted the potential of elraglusib, citing interim Phase 2 data showing statistically significant improvements in 1-year and median overall survival when combined with gemcitabine/nab-paclitaxel (GnP). The trial involves additional sites, including The University of Colorado and the University of Washington’s Fred Hutchinson Cancer Center. Dr. Weekes expressed optimism about the trial's potential to improve treatment options for mPDAC patients.

Positive

• Completion of patient enrollment in Phase 2 trial.
• Initial data shows early evidence of enhanced clinical activity.
• Interim Phase 2 data shows significant improvements in survival.
• Collaboration with top research institutions.

Negative

• Final results not expected until 2026.

Insights

Biotechnology Analyst

Actuate Therapeutics has reached a significant milestone with the completion of enrollment in its Phase 2 trial evaluating elraglusib combined with FOLFIRINOX and losartan for previously untreated metastatic pancreatic cancer (mPDAC). This development warrants investor attention given pancreatic cancer's dismal 5-year survival rate of approximately 11% and effective treatment options.

The trial's design is particularly noteworthy. FOLFIRINOX (a chemotherapy regimen combining 5-fluorouracil, leucovorin, irinotecan, and oxaliplatin) represents the current standard of care but offers modest survival benefits. The addition of elraglusib, a GSK-3β inhibitor, targets a novel pathway implicated in pancreatic cancer progression and chemoresistance. The further inclusion of losartan, traditionally an antihypertensive medication, is based on emerging research suggesting it may reduce tumor-associated fibrosis and improve drug delivery in the dense pancreatic tumor microenvironment.

Initial data presented at the AACR Special Conference showed "early evidence of enhanced clinical activity" - though specific metrics weren't disclosed, this positive language suggests potential efficacy signals. More compelling is the company's reference to a separate Phase 2 trial combining elraglusib with gemcitabine/nab-paclitaxel that demonstrated "statistically significant improvements in 1-year and median overall survival" - pursuing multiple combination approaches creates several potential paths to market.

The trial's prestigious collaborators add credibility - Massachusetts General Hospital, University of Colorado, Fred Hutchinson Cancer Center, and financial support from the Lustgarten Foundation. This institutional backing suggests scientific merit beyond typical company-sponsored research.

For investors, the extended timeline to final results (2026) creates both risk and opportunity. With a $150 million market cap, positive interim data could drive significant valuation increases before final results. However, the company will need to manage cash runway carefully through this extended development period.

If successful, elraglusib could address a global pancreatic cancer market projected to reach $4.2 billion by 2027. Given Actuate's size, positive results would likely attract partnership interest from larger oncology players seeking to expand their presence in this high-unmet-need indication.

• Initial Data from this Phase 2 Trial Demonstrates Early Evidence of Clinical Activity of Elraglusib (9-ING-41) in Combination with FOLFIRINOX and Losartan in Untreated Metastatic Pancreatic Ductal Adenocarcinoma (mPDAC)
• The Final Results from the Trial are Anticipated in 2026

CHICAGO and FORT WORTH, Texas, Feb. 25, 2025 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company, focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), announced the completion of patient enrollment in the ongoing Phase 2 trial of FOLFIRINOX in combination with elraglusib and losartan for untreated metastatic pancreatic adenocarcinoma (mPDAC). The trial is being led by Colin Weekes MD PhD at Massachusetts General Hospital and is supported by the Lustgarten Foundation as well as Actuate Therapeutics. Additional sites participating in the study include The University of Colorado (led by Cristopher Lieu, MD) and the University of Washington’s Fred Hutchinson Cancer Center (led by Andrew Coveler, MD).

The trial enrolled 56 treatment-naïve mPDAC patients. The primary objectives of this Phase 2 open-label, non-comparator study (NCT05077800) are to determine the safety, tolerability, and progression-free survival of the combination of elraglusib with FOLFIRINOX and losartan .

“The completion of enrollment in this trial represents an important milestone in the development of elraglusib in metastatic pancreatic cancer,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “Initial data from this combination trial, as presented by Dr. Weekes’ team at the AACR Special Conference on Advances in Pancreatic Cancer Research in September 2024, demonstrated early evidence of enhanced clinical activity when elraglusib was combined with FOLFIRINOX and losartan in patients with untreated mPDAC. We are proud to support this important trial and remain deeply committed to advancing elraglusib for patients with advanced cancer including mPDAC.”

Mr. Schmitt continued, “Additionally, our recently reported interim Phase 2 data of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) also in the first-line treatment of mPDAC showed statistically significant improvements in 1-year and median overall survival, highlighting the potential of combining elraglusib with several different 1^st line chemotherapy modalities in the treatment of mPDAC. We look forward to continuing both trials and further advancing our understanding of elraglusib’s therapeutic impact on mPDAC.”

“We are happy to complete patient enrollment in this important trial, which represents a significant milestone in our efforts to improve the treatment of metastatic pancreatic cancer,” said Colin Weekes, MD, Ph.D., Director of Medical Oncology Research for Pancreatic Cancer, Massachusetts General Hospital, and the study’s Principal Investigator. “Our team is optimistic that the insights gained from this trial will drive meaningful progress in developing more effective treatment options for patients. We look forward to reporting final results from the trial in 2026.”

About Actuate Therapeutics, Inc.

Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate’s lead investigational drug product, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy including several DNA Damage Response (DDR) pathways. Elraglusib is designed to act as a mediator of anti-tumor immunity through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulates multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at http://www.actuatetherapeutics.com.

About Massachusetts General Hospital
Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The Mass General Research Institute conducts the largest hospital-based research program in the nation, with annual research operations of more than $1 billion and comprises more than 9,500 researchers working across more than 30 institutes, centers and departments. MGH is a founding member of the Mass General Brigham healthcare system.

Forward-Looking Statements

This press release contains forward-looking statements about us, including our clinical trials and development plans, and our industry. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies and early clinical trials are not necessarily predictive of future results, and elraglusib may not achieve positive clinical results or favorable preclinical results or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development, including through conversations with the FDA or EMA and the standards such bodies may impose for such development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; preliminary and unpublished data may be subject to change following the availability of more data or following a more comprehensive review of the data and should not be relied upon as a final analysis; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities; and our ability to realize any benefits or value associated with the Orphan Medicinal Product Designation (OMPD) granted to elraglusib by the EMA for the treatment of pancreatic ductal adenocarcinoma (PDAC). In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading “Item 1A. Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 filed with the SEC on November 13, 2024 and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events

Investor Contact

Mike Moyer

Managing Director

LifeSci Advisors, LLC

mmoyer@lifesciadvisors.com

FAQ

What is the significance of Actuate's Phase 2 trial completion for ACTU stock?

The completion of patient enrollment in Actuate's Phase 2 trial is a major milestone, indicating progress in developing elraglusib for metastatic pancreatic cancer, which could positively impact ACTU stock.

When are the final results of the ACTU Phase 2 trial expected?

The final results of Actuate's Phase 2 trial are anticipated in 2026.

What initial data has been observed in Actuate's Phase 2 trial?

Initial data from Actuate's Phase 2 trial showed early evidence of enhanced clinical activity when combining elraglusib with FOLFIRINOX and losartan in untreated mPDAC patients.

How many patients were enrolled in Actuate's Phase 2 trial?

Actuate's Phase 2 trial enrolled 56 treatment-naïve mPDAC patients.

What are the primary objectives of Actuate's Phase 2 trial?

The primary objectives of Actuate's Phase 2 trial are to determine the safety, tolerability, and progression-free survival of the combination therapy.

What other trials are ongoing with elraglusib in mPDAC?

Actuate is also conducting a Phase 2 trial of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) for first-line treatment of mPDAC.